Unmet Medical Needs Research Articles

HPR209 The Unmet Medical Needs Concept – A Structured Analysis of Its Application in Authorisation and Belgian Reimbursement Practices

Unmet Medical Need (UMN) is a criterion for specific regulatory and market access procedures that are in place to accelerate access to highly needed medicines. As the interpretation of the concept differs among stakeholders, this study aims to (i) identify the criteria by which an UMN is characterised, (ii) identify the type of criteria reflected upon in (European) regulatory and (national) market access documents and the extent to which they are discussed, and (iii) identify differences and similarities between regulatory and market access documents in the reporting of UMN.

Unmet Medical Needs and Food Insecurity in Children with Neurodevelopmental Disorders: Findings from the 2019 National Health Interview Survey (NHIS).

In the United States, 17% of children ages 3-17 have a developmental disorder. The complexity of care for such children require families to provide a significant amount of health care at home, representing a substantial economic cost. Our study identifies sociodemographic characteristics of children with neurodevelopmental disorders (NDD) that are predictive of unmet medical needs and food insecurity. We modeled the outcomes using a multivariable generalized linear model and a robust Cox proportional hazard model. Among children with NDD, 7.4% reported a delay in obtaining care, 3.6% avoided getting care and 17.3% live in a household that experienced food insecurity. Lack of health insurance and lack of usual source of care increased the risk for cost-related delay in medical care and cost-related avoidance of medical care. Children with NDD whose parents have less than a college degree and those from households with income <$75,000 had increased risk for food insecurity in the past 30 days. Our results underscore the need to implement additional screening to identify children with NDD who are at greater risk for unmet medical and social needs by health care providers and care coordination organizations.

Unmet Medical Needs and Early Referral of Pediatric Atopic Dermatitis: An Expert Modified Delphi Consensus from Saudi Arabia.

Atopic dermatitis (AD) is a chronic skin disease with increasing prevalence worldwide. It is characterized by pruritic eczematous lesions, affecting up to 20% of the children and negatively impacting their quality of life. Guidelines for AD management are available worldwide, but specific guidelines for pediatric AD in Saudi Arabia are lacking. This consensus document aims to identify the needs for the diagnosis and management of pediatric AD in Saudi Arabia by gathering the opinions and recommendations of key experts. We conducted a three-step modified Delphi method to develop the present consensus. The experts agreed that pediatricians and dermatologists commonly encounter AD; however, it is still under-recognized in its early stage in Saudi Arabia. The family physicians should be involved in assessing suspected children with a family history of atopy, particularly in patients with isolated lesions. Further, the experts confirmed that AD diagnosis should be documented, showing assessment criteria used, key morphological characteristics, and features used to ascertain the severity of the disease. There is still a need for simple validated diagnostic criteria suitable for daily practice for pediatric AD. The experts highlighted several medical conditions that pertain to the diagnosis and management of AD in Saudi Arabia.

Unmet Medical Needs in Chronic, Non-communicable Inflammatory Skin Diseases.

An estimated 20–25% of the population is affected by chronic, non-communicable inflammatory skin diseases. Chronic skin inflammation has many causes. Among the most frequent chronic inflammatory skin diseases are atopic dermatitis, psoriasis, urticaria, lichen planus, and hidradenitis suppurativa, driven by a complex interplay of genetics and environmental factors. Autoimmunity is another important cause of chronic skin inflammation. The autoimmune response may be mainly T cell driven, such as in alopecia areata or vitiligo, or B cell driven in chronic spontaneous urticaria, pemphigus and pemphigoid diseases. Rare causes of chronic skin inflammation are autoinflammatory diseases, or rheumatic diseases, such as cutaneous lupus erythematosus or dermatomyositis. Whilst we have seen a significant improvement in diagnosis and treatment, several challenges remain. Especially for rarer causes of chronic skin inflammation, early diagnosis is often missed because of low awareness and lack of diagnostics. Systemic immunosuppression is the treatment of choice for almost all of these diseases. Adverse events due to immunosuppression, insufficient therapeutic responses and relapses remain a challenge. For atopic dermatitis and psoriasis, a broad spectrum of innovative treatments has been developed. However, treatment responses cannot be predicted so far. Hence, development of (bio)markers allowing selection of specific medications for individual patients is needed. Given the encouraging developments during the past years, we envision that many of these challenges in the diagnosis and treatment of chronic inflammatory skin diseases will be thoroughly addressed in the future.

Disease Impact, Diagnostic Delay, and Unmet Medical Needs of Patients With Cholinergic Urticaria in German-Speaking Countries.

BackgroundCholinergic urticaria (CholU) is a common type of chronic inducible urticaria. Little is known about the burden of the disease and its unmet medical needs.AimTo characterize the unmet medical needs of patients with CholU.MethodsPatients with CholU (n = 111) took part in a German online survey that assessed their symptoms, diagnostic delay, impact on daily life, quality of life (QoL), and their experience with physician care.ResultsVirtually all patients reported typical signs and symptoms of CholU, i.e., whealing (93.7%) and itching (91.9%), in response to typical trigger situations, such as physical activity, passive warming, or stress. Despite this, patients reported a marked diagnostic delay of 30.2 months (range from 0 to 279 months). Only 38% of the patients received a blood examination, and only 16% underwent provocation testing for diagnosing CholU, as recommended by the international guidelines. Physician contacts were common, but patient satisfaction with their disease management was low. In total, 90.1% of the patients stated to have an uncontrolled disease, resulting in a strong impact on their everyday activities, sleep, and QoL.ConclusionPatients with CholU exhibit many important unmet needs, and improvement in the diagnostic workup and patient care is needed, as are better treatment options.

「不」に応える：DDSの臨床応用と事業化の推進

DDS創薬は既存原薬に対して、臨床効果、経済効果など、圧倒的優位性を提供できる。優位性を実現するには、臨床開発戦略と薬価を含む事業化戦略との切磋琢磨、整合性が欠かせない。筆者らは、シーズ思考およびニーズ思考と2つの異なる視点から製品像TPPをデザインする。患者、医師、パラメディカル、行政当局、保険者、製薬企業にとって6方よしを実現する前提で、当該製品の事業価値を最大化するよう、R&D/事業化戦略部門がインタラクションしている。本稿では、当該適応症における患者数、薬価、製造原価、R&Dコスト、営業利益を含む定量予測モデルの概念を紹介している。

COVID-19 and Unmet Medical Needs for People With Chronic Diseases: A Cross-Sectional Study.

Gaps between medical needs and health services accessibility are defined as unmet medical needs (UMN). Previous studies assessing UMNs during the coronavirus disease 2019 (COVID-19) pandemic did not account for factors such as the type of medical insurance or chronic disease. Therefore, this study investigated various factors, including medical insurance and chronic diseases, affecting self-reported UMN during the pandemic in Daejeon Metropolitan City, South Korea. This cross-sectional study was conducted using Daejeon Metropolitan City Health Surveys data from 2020. The weighted prevalence of UMN was estimated among participants (n = 1494) aged ≥18 years, while chi-squared and logistic models were used to assess the association between UMN and participant characteristics. The prevalence of UMN was 6.4% (n = 97). Female sex and low education levels were significantly associated with reporting UMN. Medical aid beneficiaries were less likely to report UMN than adults with National Health Programs coverage. Participants with chronic diseases, such as hypertension and arthritis, and those with depression, were more likely to report UMN than adults without these conditions. The findings highlight the need for targeted efforts to decrease UMN in the COVID-19 pandemic, especially for women and individuals with low education levels. The results also indicate that high-risk disease groups should be targeted with additional enabling services to support the management of chronic conditions.

Systems Biology to Address Unmet Medical Needs in Neurological Disorders.

Neurological diseases are highly prevalent and constitute a significant cause of mortality and disability. Neurological disorders encompass a heterogeneous group of neurodegenerative conditions, broadly characterized by injury to the peripheral and/or central nervous system. Although the etiology of neurological diseases varies greatly, they share several characteristics, such as heterogeneity of clinical presentation, non-cell autonomous nature, and diversity of cellular, subcellular, and molecular pathways. Systems biology has emerged as a valuable platform for addressing the challenges of studying heterogeneous neurological diseases. Systems biology has manifold applications to address unmet medical needs for neurological illness, including integrating and correlating different large datasets covering the transcriptome, epigenome, proteome, and metabolome associated with a specific condition. This is particularly useful for disentangling the heterogeneity and complexity of neurological conditions. Hence, systems biology can help in uncovering pathophysiology to develop novel therapeutic targets and assessing the impact of known treatments on disease progression. Additionally, systems biology can identify early diagnostic biomarkers, to help diagnose neurological disease preceded by a long subclinical phase, as well as define the exposome, the collection of environmental toxicants that increase risk of certain neurological diseases. In addition to these current applications, there are numerous potential emergent uses, such as precision medicine.

Directly and Indirectly Targeting the Glycine Modulatory Site to Modulate NMDA Receptor Function to Address Unmet Medical Needs of Patients With Schizophrenia.

Schizophrenia is a severe mental illness that affects ~1% of the world's population. It is clinically characterized by positive, negative, and cognitive symptoms. Currently available antipsychotic medications are relatively ineffective in improving negative and cognitive deficits, which are related to a patient's functional outcomes and quality of life. Negative symptoms and cognitive deficits are unmet by the antipsychotic medications developed to date. In recent decades, compelling animal and clinical studies have supported the NMDA receptor (NMDAR) hypofunction hypothesis of schizophrenia and have suggested some promising therapeutic agents. Notably, several NMDAR-enhancing agents, especially those that function through the glycine modulatory site (GMS) of NMDAR, cause significant reduction in psychotic and cognitive symptoms in patients with schizophrenia. Given that the NMDAR-mediated signaling pathway has been implicated in cognitive/social functions and that GMS is a potential therapeutic target for enhancing the activation of NMDARs, there is great interest in investigating the effects of direct and indirect GMS modulators and their therapeutic potential. In this review, we focus on describing preclinical and clinical studies of direct and indirect GMS modulators in the treatment of schizophrenia, including glycine, D-cycloserine, D-serine, glycine transporter 1 (GlyT1) inhibitors, and D-amino acid oxidase (DAO or DAAO) inhibitors. We highlight some of the most promising recently developed pharmacological compounds designed to either directly or indirectly target GMS and thus augment NMDAR function to treat the cognitive and negative symptoms of schizophrenia. Overall, the current findings suggest that indirectly targeting of GMS appears to be more beneficial and leads to less adverse effects than direct targeting of GMS to modulate NMDAR functions. Indirect GMS modulators, especially GlyT1 inhibitors and DAO inhibitors, open new avenues for the treatment of unmet medical needs for patients with schizophrenia.

EPICOVIDEHA: A Ready to Use Platform for Epidemiological Studies in Hematological Patients With COVID-19

EPICOVIDEHA: A Ready to Use Platform for Epidemiological Studies in Hematological Patients With COVID-19

Perceived Discrimination and Increased Odds of Unmet Medical Needs Among US Children.

Our study examines the association between perceived discrimination due to race and unmet medical needs among a nationally representative sample of children in the United States. We used data from the 2016-2017 National Survey of Children's Health, a population-based cross-sectional survey of randomly selected parents or guardians in the United States. We compared results from the coarsened exact matching (CEM) method and survey-weighted logistic regression to assess the robustness of the results. Using self-reported measures from caregivers, we find that ∼2.7% of US children have experienced racial discrimination with prevalence varying significantly by race. While <1% of non-Hispanic whites have experienced some measure of racism, this increases to 8.8% among non-Hispanic blacks. Perceived discrimination was associated with significantly greater odds of unmet medical needs in the adjusted, survey-weighted multivariate-adjusted model (adjusted odds ratio [OR] = 2.4 and 95% confidence interval [CI] = 1.2, 4.9) as well as in the CEM-model estimate (OR = 2.8 and 95% CI = 1.8, 4.0). Children who have experienced perceived discrimination had higher odds of unmet medical needs. Awareness of discrimination among children may help inform future intervention development that addresses unmet medical needs during childhood.

Unmet Medical Needs in the Treatment and Management of Generalized Pustular Psoriasis Flares: Evidence from a Survey of Corrona Registry Dermatologists.

IntroductionGeneralized pustular psoriasis (GPP) is a rare, severe, and potentially life-threatening systemic and chronic autoinflammatory disease characterized by sterile, neutrophilic pustules. The standard of care for GPP varies by region, with limited information and experience of flares and their treatment. Our aim was to establish current unmet needs in GPP by better understanding the natural history of GPP, examining how dermatologists diagnose GPP and GPP flares, and establishing the range and adequacy of GPP treatment options currently prescribed by dermatologists.MethodsEligible dermatologists (N = 29) completed a 28-question structured survey, covering ten themes, ranging from GPP diagnostic criteria to GPP symptoms and treatment.ResultsAll dermatologists stated that pustules were necessary to diagnose a GPP flare. The most frequently reported triggering factors for GPP were steroid withdrawal (64%), infection (58%), and stress (50%). Most dermatologists indicated that available treatment options for GPP flares were adequate “most” (79%) or “all” (14%) of the time. Despite this reported adequacy, 38% of dermatologists reported that it was at least “somewhat common” for a flare to require hospitalization. Furthermore, 72% of dermatologists indicated that treatments were too slow to control flares, and 66% indicated that treatments did not adequately prevent new flares at least “sometimes”.ConclusionThis survey suggests that there are key features of GPP flares, and could initiate discussion around forming consensus guidelines for diagnosis and management. While the results suggest that moderately effective therapies may exist, the need for GPP-specific treatments remains.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13555-021-00493-0.

Unmet Medical Needs and Future Perspectives for Leiomyosarcoma Patients-A Position Paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN).

Simple SummaryIn this position paper, we aim to summarize state-of-the-art treatments for patients with leiomyosarcomas in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This white paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN).As leiomyosarcoma patients are challenged by the development of metastatic disease, effective systemic therapies are the cornerstone of outcome. However, the overall activity of the currently available conventional systemic treatments and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of this patient group challenging. Therefore, in a joint effort together with patient networks and organizations, namely Sarcoma Patients EuroNet (SPAEN), the international network of sarcoma patients organizations, and the National LeioMyoSarcoma Foundation (NLMSF) in the United States, we aim to summarize state-of-the-art treatments for leiomyosarcoma patients in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This position paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the NLMSF and SPAEN.

Unmet Medical Needs Among Adults Who Move due to Unaffordable Housing: California Health Interview Survey, 2011-2017.

BackgroundStable, affordable housing is an established determinant of health. As affordable housing shortages across the USA threaten to displace people from their homes, it is important to understand the implications of cost-related residential moves for healthcare access.ObjectiveTo examine the relationship between cost-related moves and unmet medical needs.DesignWe performed a cross-sectional analysis of 7 waves (2011–2017) of the California Health Interview Survey.ParticipantsWe included all respondents ages 18 and older.Main MeasuresThe primary predictor variable was residential move history in the past 5 years (cost-related move, non-cost-related move, or no move). The primary outcome was unmet medical needs in the past year (necessary medications and/or medical care that were delayed or not received).Key ResultsOur sample included 146,417 adults (42–47% response rate), representing a weighted population of 28,518,590. Overall, 20.3% of the sample reported unmet medical needs in the past year, and 4.9% reported a cost-related move in the past 5 years. In multivariable logistic regression models, adjusted risk of unmet medical needs increased for adults with both cost-related moves (aOR 1.38; 95% CI 1.19–1.59) and non-cost-related moves (aOR 1.17; 95% CI 1.09–1.26) compared to those with no moves. Among people who had moved, those with cost-related moves were more likely to report unmet medical needs compared to people with non-cost-related moves (p = 0.03).ConclusionsPeople who have moved due to unaffordable housing represent a population at increased risk for unmet medical needs. Policy makers seeking to improve population health should consider strategies to limit cost-related moves and to mitigate their adverse effects on healthcare access.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11606-020-06347-3.

The Effects of Social Networks of the Older Adults with Limited Instrumental Activities of Daily Living on Unmet Medical Needs.

This study was conducted to identify the effects of social networks on unmet medical needs among older adults with limited instrumental activities of daily living (IADL) who live in a community. This study analyzed data from 2281 older adults with limited IADL from the 2017 National Survey of Older Koreans. Data were analyzed using descriptive statistics, X2 tests, t-tests, and logistic regression analysis. About 73.0% of the subjects were female and 15.8% of the subjects had experienced unmet medical needs. The predictors of unmet medical needs according to gender are as follows: annual household income, participation in social activities, and physical support for male subjects and annual household income, number of chronic diseases, living alone in a household, living with others in a household, frequency of contacting close friends, and emotional support for female subjects. The findings of this study will be utilized as a basis for establishing relevant measures to enable older adults to receive proper medical services by heightening the understanding of the gap between medical service use and the medical needs of older adults with limited IADL.

Drug Prices, Dying Patients, and the Pharmaceutical Marketplace: A New Conditional Approval Pathway for Critical Unmet Medical Needs

Prescription drugs are much in the news these days. There are two topics which appear very often; first, the very high prices of new drugs, particularly the “specialty” drugs developed for serious diseases; and second, the time required for FDA approval in relation to the perceived need for earlier access to new therapies for critically ill patients. Much less in the news, but lurking behind both problems, is the need for better information for physicians and patients to use in making decisions about prescribing and taking drugs and for insurance companies and the government to use to structure their pharmaceutical benefits plans. This article proposes an approach to both accelerated access and drug prices that would generate the information doctors and patients need to make decisions about drugs and the information the government and private insurers need to provide appropriate access and coverage for those drugs. The new form of conditional approval proposed here would be similar to the parallel track program developed by the FDA in the 1990s, during the HIV crisis. I argue that, like parallel track, the conditional approval proposed here could be implemented by the FDA under its existing authority, allow wide access to desperately needed drugs, and would control prices while providing better information about the real safety and efficacy of new drugs for critically ill patients.

The Unmet Medical Needs of Current Injectable Antidiabetic Therapies in China: Patient and Health Care Professional Perspectives

PurposePatients with diabetes and health care professionals (HCPs) play important roles in effective application of injectable antidiabetic therapies (IATs). However, their concerns and opinions on IATs are rarely investigated in China. This study aims to assess unmet medical needs of IATs regarding patient concerns, patient satisfaction, aspects that need improvement, and training burden from patient and HCP perspectives. MethodsThis cross-sectional survey was conducted in 12 representative Chinese cities from December 2018 to January 2019. Patients with adult type 2 diabetes who were receiving IAT currently and had received IAT continuously for at least 1 month before the survey, endocrinologists with ≥5 years of experience and prescribing IAT in the past 1 month, and nurses with ≥3 years of experience and providing IAT training in the past 1 month were eligible participants. The patient survey assessed concerns of initiating IAT, satisfaction with IAT, aspects of IAT that need improvement, and IAT training received. The HCP survey evaluated patient concern of initiating IAT, aspects of IAT that need improvement, experience of providing IAT training, and self-reported burden of training. Descriptive statistical analysis was performed. FindingsIn total, 500 patients, 200 endocrinologists, and 100 nurses were surveyed. The mean (SD) age of patients was 55.1 (11.8) years, with a disease duration of 7.6 (6.4) years. Of all patients, 391 (78.2%) were insulin users and 109 (21.8%) were glucagon-like peptide 1 receptor agonist users. Of the top 4 concerns about initiating IAT, both patients and endocrinologists reported inconvenience of daily injection (58.0% of patients and 68.5% of endocrinologists), worries about insulin dependence (42.6% of patients and 62.5% of endocrinologists), and fear of injection (37.0% of patients and 66.5% of endocrinologists). Medical expenses, convenience of drug portability and storage, and injection site reactions were the top 3 aspects that need improvement according to both patients and HCPs. High injection frequency was also one of the most urgent aspects for improvement (mean urgency score, 3.8 for physicians and 4.0 for nurses). A typical IAT training session took a mean (SD) of 14.1 (9.7) minutes. Both patients and HCPs considered injection operation after dose is set and symptoms and treatment for adverse effects as the 2 most time-consuming training contents. In addition, 97.1% of endocrinologists who provided training and 97.0% of nurses thought a more user-friendly IAT would reduce their training burden. ImplicationsStudy results indicate that the IATs with more convenient drug portability and storage, fewer injection site reactions and adverse events, less injection frequency, more user-friendly design, and fewer steps for injection might help improve patient experience with self-injection and reduce HCPs’ training burden.

의료보장유형별 미충족의료에 영향을 미치는 요인에 관한 다수준 분석

의료보장유형별 미충족의료에 영향을 미치는 요인에 관한 다수준 분석

Toward a DDS Research Center for Targeting Unmet Medical Needs

Toward a DDS Research Center for Targeting Unmet Medical Needs

PDB108 THE UNMET MEDICAL NEEDS IN CURRENT ANTIDIABETIC INJECTABLE THERAPY (AIT) IN CHINA: FROM THE PERSPECTIVE OF HEALTHCARE PROVIDERS (HCPS)

This study aimed to understand HCP perceptions of AIT for type 2 diabetes mellitus (T2DM). This is a part of the PURPOSE study (Patient, Nurse, and Physician Survey on Unmet Medical Needs of Injectable Diabetes Treatments), a cross-sectional survey conducted in 13 representative cities across China in 2019. 200 endocrinologists prescribing at least 10 prescriptions of AIT and 100 nurses providing AIT training in the past month were surveyed. Questionnaires focused on reasons for which patients rejected AIT, areas of AIT requiring improvement, factors influencing patient compliance, and HCP’s AIT training burden. The extent of influence of each factor on compliance was measured with a 5-point scale (1=no influence, 5=huge influence). Descriptive statistics were conducted using R software, version 3.5.2. 81.0% of endocrinologists had 10+ years of experience and 64.0% held deputy chief titles or above. 85.0% of nurses had 5+ years of experience. 97.0% of endocrinologists reported they had patients who refused to initiate AIT. Major concerns about AIT initiation included inconvenience of daily injection (68.5%), fear of injection (66.5%), and feelings that injection indicates serious disease status (63.0%). Injection site reactions, medical expenses, and inconvenience of drug portability/storage were considered the first three areas requiring improvement by endocrinologists (69.5%, 60.0%, 56.0%, respectively) and nurses (70.0%, 54.0%, 52.0%, respectively). The top three influential factors of compliance rated by endocrinologists were injection frequency (3.8), severity of diabetes (3.8), and injection training (3.8). Injection operation, side effects management, and pre-injection dose setting were considered the most time-consuming components in a typical AIT training. 97.1% of endocrinologists and 97.0% of nurses believed a more user-friendly product would reduce training burden. From a HCP’s perspective, AIT training and AIT with less injection frequency will help improve patient compliance. A more user-friendly AIT with less side effects would help reduce HCP’s training burden.